9 Meters Biopharma Inc., of Raleigh, N.C., said it closed its merger with Naia Rare Diseases Inc., of Richmond, Calif., which develops a long-acting GLP-1 receptor agonist to treat short bowel syndrome and other rare gastrointestinal diseases. The deal also includes a long-acting GLP-2 candidate being developed for an undisclosed orphan indication. The transaction follows a recent financing, led by Orbimed Advisors, for $22 million in new funding.
AI Vivo, of Cambridge, U.K., which combines systems pharmacology and artificial intelligence (AI) to accelerate drug discovery, said it is seeking pharma and biotech collaboration partners to progress therapeutic candidates that have been identified by AI Vivo as “top-ranked” for COVID-19. The announcement follows the finding that 41 of the candidate drugs from the company’s top-ranked list for COVID-19 are now in clinical trials by multiple groups. Other candidates identified by the platform provide antiviral, anti-inflammatory and cytokine storm suppressive effects, the company said.
Amryt Pharma plc, of London, said the global brand name for its lead development product, AP-101, will be Filsuvez. The drug is in development as a potential treatment for epidermolysis bullosa.
Aum Lifetech Inc., of Philadelphia, and Aum Biotech LLC, also of Philadelphia, a genetic research startup, have jointly launched a research program called Knockdown Coronavirus with a goal of developing a treatment for COVID-19. Under this initiative, the two companies are offering self-delivering RNA-silencing research products to the global coronavirus research community to facilitate research and fast track therapeutic development. Aum Biotech said it RNA-targeting technology can be used to perform high-throughput genetic screening to identify the function of viral and host genes and help identify new targets for COVID-19 therapy development, the companies said.
Azurrx Biopharma Inc., of New York, signed an agreement with Creapharm Group, of Reims, France, for the packaging, labeling and distribution of phase II trial drug product supply of its MS-1819 therapy for the treatment of exocrine pancreatic insufficiency (EPI). Creapharm will handle drug product for both the upcoming phase IIb Option 2 monotherapy study and the ongoing phase II combination therapy trial for the treatment of EPI in patients with cystic fibrosis. MS-1819 will be sent directly to clinical sites in the U.S. and in Europe, including Poland, Hungary, Spain and Turkey, from Creapharm’s U.S. and European depots, respectively.
Betterlife Pharma Inc., of Vancouver, British Columbia, said it entered an agreement to acquire worldwide rights (other than in greater China, Japan and ASEAN countries) to commercialize and sell Anticovir, a potential COVID-19 treatment, from Altum Pharmaceuticals Inc., also of Vancouver. Anticovir is an interferon a2b-based potential treatment that is proposed to be administered using a metered dose inhaler or a nebulizer. Altum is currently preparing the protocol and application to conduct a 306-patient randomized, double-blind, placebo-controlled phase III registrational trial in Australia. Subject to regulatory approvals, trials in Australia could begin as early as July 2020. Under the terms of the transaction, Betterlife will issue on closing 10 million common shares to Altum and grant to Altum 5 million warrants to acquire an equivalent number of common shares at a price of CA19 cents (US14 cents) per common share. In addition, subject to the satisfaction of certain conditions precedent, upon registration of the proposed product in a major market, Betterlife will pay CA$5 million in cash to Altum, and Altum will be entitled to a tiered royalty equal to 7% of net sales on the first CA$50 million in a calendar year and a reduced royalty equal to 5% of net sales in any calendar year that are in excess of CA$50 million. Betterlife has, subject to raising the necessary funds, agreed to fund the first $15 million of costs required for the proposed phase III trials.
Biontech SE, of Mainz, Germany, closed the acquisition of Neon Therapeutics Inc., of Cambridge, Mass., by way of an all-stock deal. The new subsidiary will operate under the name of Biontech U.S. Inc. and serve as U.S. headquarters. Neon’s common stock is no longer available for trading.
Caribou Biosciences Inc., of Berkeley, Calif., and Maxcyte Inc., of Gaithersburg, Md., disclosed a clinical and commercial license agreement. Under the terms, Caribou gains rights to use Maxcyte's Flow Electroporation technology and ExPERT platform for the advancement of its CRISPR gene-edited allogeneic T-cell therapy programs. Maxcyte will receive undisclosed development and approval milestone payments and sales-based rewards plus other licensing fees.
Certara Inc., of Princeton, N.J., and Australia’s DMTC (formerly the Defense Materials Technology Center) Ltd. signed a research collaboration to evaluate the preventive use of chloroquine in health care workers at risk of infection from the coronavirus strain that causes COVID-19. Certara will support the DMTC-led clinical trial by analyzing study data to develop mathematical models to understand the viral time course and factors impacting drug exposure and prophylactic response in health care workers. DMTC’s Chloroquine Chemoprophylaxis Countermeasure Against COVID-19 study is a randomized, controlled study of healthy, adult health care workers drawn from military and civilian workforces at risk of COVID-19 infection. Participation in the trial is entirely voluntary, subject to fully informed consent and strict admittance criteria.
Cidara Therapeutics Inc., of San Diego, reported preclinical data in abstracts published by the European Congress of Clinical Microbiology and Infectious Diseases from a rabbit model of Candida endophthalmitis, showing treatment with rezafungin significantly lowered fungal burden as compared to micafungin, an echinocandin approved for the treatment of Candida infection, voriconazole, an azole for the treatment of Candida infection, and vehicle control in multiple tissues of the eye. The rezafungin-treated group was the only one to demonstrate no eye lesions following treatment. Published abstracts also detailed findings from Cidara’s Cloudbreak antiviral candidate, CD-377, for the prevention and treatment of influenza, including in vitro and in vivo data showing it was stable and intact after incubations in mouse and human plasma and human hepatocytes. Single doses of CD-377 in a lethal mouse model of influenza A showed a greater decrease in lung viral burden and cytokine levels compared to oseltamivir dosed for five days, twice daily at 10 times the human equivalent dose. Additional preclinical data showed that mice treated with single, low doses of CD-377 were protected in lethal challenge models with several seasonal influenza subtypes. A single CD-377 dose resulted in complete recovery from lethal challenge with several influenza strains.
The CoVIg-19 Plasma Alliance, a plasma industry collaboration recently established to accelerate the development of a plasma-derived hyperimmune globulin therapy for COVID-19, has expanded globally to include 10 plasma companies, and now also includes global organizations from outside the plasma industry. All are providing support to encourage more people to donate plasma. They will contribute specialist advisory expertise, technical guidance and/or in-kind support to contribute to the alliance’s goal of accelerating development and distribution of a potential treatment option for the virus. Latest industry members to join include Adma Biologics Inc., of Ramsey, N.J.; Biopharma Plasma LLC, of Kryvyi Rih, Ukraine; GC Pharma, of Gyeonggi-do, South Korea; and Sanquin, of Amsterdam.
Errant Gene Therapeutics Inc. (EGT), of Chicago, said that on April 30, the New York Supreme Court, First Department, Appellate Division unanimously affirmed rulings in EGT’s favor to proceed with fraud, unfair competition and breach of contract claims in a trial scheduled Oct. 29, 2020, against Bluebird Bio Inc., of Cambridge, Mass., and Sloan Kettering Institute for Cancer Research (SKI). EGT alleged that SKI secretly disclosed EGT’s confidential information to Bluebird in furtherance of a scheme to shelve EGT’s gene therapy and to exploit EGT’s intellectual property so that Bluebird could develop and commercialize its own gene therapy. In June 2019, Bluebird Bio (now absent competition) raised the price of its therapy to $1.8 million per patient, according to EGT.
Frame Therapeutics BV, of Amsterdam, and two clinical parties from the university medical centers in Amsterdam and Groningen make up a consortium with a total research budget of €1.2 million (US$1.29 million). The group has received the Kansen voor West grant to cover 40% of the project named Framome. Kansen voor West is a subsidy that is part of the European Regional Development Fund, which stimulates regional economies and innovative collaborations between small and medium-sized businesses, knowledge institutions and government bodies. The consortium combines knowledge on bioinformatics, big data analysis and genome sequencing with clinical expertise, specifically in pancreatic and lung oncology.
Idbiologics Inc. and Vanderbilt University Medical Center, both of Nashville, Tenn., have agreed to work together to develop monoclonal antibodies (Mabs) for COVID-19 treatment and prevention. Backed by the U.S. Defense Advanced Research Projects Agency, the NIH and the Dolly Parton COVID-19 Research Fund at Vanderbilt, the Vanderbilt Vaccine Center (VVC) researchers have developed the ability to identify and isolate individual B cells that produce antibodies that target a specific viral protein, they said. Vanderbilt will now share MAbs discovered in the VVC with Idbiologics for evaluation and potential licensing and clinical development.
Iontas Ltd., of Cambridge, U.K., signed a collaboration agreement with Inotrem SA, of Paris, for acute and chronic inflammatory syndromes. Iontas will apply its mammalian display technology to identify antibodies involved in the TREM-1 pathway. TREM-1, an immunoreceptor expressed on innate immune cells, has been identified by Inotrem as a key therapeutic target for many acute or chronic inflammation syndromes, including sepsis and myocardial infarction. Iontas’ platform will enable specific high-affinity antibodies with optimal biophysical properties to be selected, to support the company in developing TREM-1 inhibition-based therapies more efficiently, with reduced chemistry, manufacturing and control risk, the companies said.
Lineage Cell Therapeutics Inc., of Carlsbad, Calif., said Cancer Research UK’s Commercial Partnerships permitted Lineage to conduct an early exercise of its option to acquire data from Cancer Research UK’s ongoing phase I trial with VAC-2 in non-small-cell lung cancer and develop an allogeneic dendritic cell therapy platform. Lineage will assume responsibility for further development of the VAC-2 product candidate as well as future development opportunities derived from the platform, while Cancer Research UK concludes the ongoing trial. The decision was based on an early review of the data collected by Cancer Research UK in the VAC-2 trial under a clinical trial and option agreement.
Lipidor AB, of Stockholm, said it will self-finance a phase III trial of AKP-02, a calcipotriol and betamethasone combo, for treating mild to moderate psoriasis. With data from a self-financed phase III study, the company said a large portion of risk is eliminated from the project and leading to a potentially yet significantly higher value for out-licensing at the next stage. The company timetable outlines the study initiation in the second quarter of 2020, with results in the first quarter of 2022.
The Lundquist Institute, of Los Angeles, began a randomized, double-blind, placebo-controlled trial designed to find out whether hydroxychloroquine and azithromycin reduce hospitalization and/or death in COVID-19 patients. The trial, sponsored by the NIH, will include 2,000 adults with symptomatic SARS-CoV-2 infections. The Lundquist Institute is one of 25 to 30 sites nationwide, each looking to enroll about 100 patients over the next six weeks. Patients will receive seven days of treatment with drugs or placebo, with a follow-up appointment at 20 days. A randomized selection of participants will receive oral doses of hydroxychloroquine or placebo twice daily for seven days, as well as a daily dose of azithromycin or placebo for five days. The initial endpoint of the study would be to evaluate the levels of hospitalization and/or death within 20 days following enrollment in the study.
Mediolanum Farmaceutici SpA, of Milan, Italy, said it acquired Elsalys Biotech SA, of Lyon, France, an immuno-oncology company working on antibodies targeting tumors and their immune and vascular environments. Financial terms were not disclosed.
Molecular Partners AG, of Zurich-Schlieren, Switzerland, said it completed in vitro potency assessments of its Darpin candidates targeting live and replicating SARS-CoV-2. The candidates show extremely robust antiviral activity, with several candidates demonstrating complete neutralization with low picomolar potency, the company added, suggesting only very small amounts of those candidates may be required for therapeutic effect. The Darpin candidates are half-life-extended and contain three distinct monomer Darpin proteins that can simultaneously target the virus in different key areas, leading to cooperative binding.
Noveome Biotherapeutics Inc., of Pittsburgh, said it received a $4 million grant from the Commonwealth of Pennsylvania to continue clinical trials of its lead product candidate, ST-266, in a wide range of diseases, including multiple ophthalmic indications. The funding will enable Noveome to shift its existing resources to support the company’s recently launched COVID-19 program evaluating ST-266 as a treatment for cytokine storm, the severe inflammatory response often observed in COVID-19 infections. ST-266 is a cell-free biologic made by culturing a population of cells derived from full-term placentas donated after a birth.
Orchard Therapeutics plc, of London, a gene therapy firm, disclosed in its first-quarter earnings a new strategic plan prioritizing metachromatic leukodystrophy candidate OTL-200, Wiskott-Aldrich syndrome drug OTL-103, mucopolysaccharidosis (MPS) type 1 drug OTL-203 and MPS-IIIA drug OTL-201 as top near-term priorities, while reducing investment in OTL-101, which targets adenosine deaminase severe combined immunodeficiency, and OTL-300, which targets transfusion-dependent beta-thalassemia. Orchard also said it plans to accelerate research in less rare indications, including two new programs in genetic subsets of frontotemporal dementia and Crohn’s disease. The company plans to focus its manufacturing strategy by prioritizing investments in technology and process innovations, closing the company’s California site, including the termination of the Fremont project and associated capital, and phasing investment in future manufacturing capacity. As a result of those decisions, Orchard has reduced its headcount by about 25%. It expects to achieve cash savings of about $125 million through the end of 2021, extending the cash runway into 2022. Shares of Orchard (NASDAQ:ORTX) closed May 7 at $10.28, down $1.62, or 13.6%.
Passage Bio Inc., of Philadelphia, expanded its collaboration agreement with the University of Pennsylvania to include an additional five programs and extending Passage Bio’s period to exercise new programs through 2025. Additionally, Passage Bio will fund discovery research at the university’s gene therapy program and receive exclusive rights, subject to certain limitations, to technologies resulting from the discovery program for Passage Bio products developed with the program, such as capsids, toxicity reduction technologies, and delivery and formulation improvements.
Rafael Pharmaceuticals Inc., of Cranbury, N.J., said it entered a research collaboration with a major medical institution to evaluate the efficacy of CPI-613 (devimistat) in combination with chemotherapy agents azacitidine and venetoclax on acute myeloid leukemia models. CP-613 targets enzymes involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells. Devimistat is designed to target the mitochondrial tricarboxylic acid cycle.
New data from the Reneuron Group plc, of London, show implantation of its CTX human neural stem cells into a model of Huntington's disease can reduce inflammation, glial scar formation and induce host neurogenesis leading to a recovery in behavioral deficits. The company said the cells can differentiate into medium spiny neurons, engraft into host tissue and form functional connections with the surrounding tissue. The company's lead clinical-stage candidates are being developed to treat retinitis pigmentosa.
Sosei Heptares Corp., of Tokyo and London, said it solved the structure of the agonist bound orexin OX2 receptor and identified a small-molecule binding site. The insights into the receptor's structure will help optimize discovery and development of molecules targeting neurological diseases, the company said. The orexin system is a key regulator of behavioral arousal, wakefulness and sleep. The loss of orexin neurons causes narcolepsy and has been linked to multiple neurological conditions.
Tonix Pharmaceuticals Holding Corp., of New York, and the University of Alberta formed a new research collaboration and exclusive licensing agreement for three new vaccines for the prevention of COVID-19. The potential vaccines, TNX-1810, TNX-1820 and TNX-1830, will be based on a horsepox vector platform but designed to express different SARS-CoV-2 antigens than TNX-1800, which is designed to express SARS-CoV-2 Spike protein, Tonix said. Under the terms of the agreement, the university granted Tonix an exclusive license for technology and patents related to the candidates.